The FDA released a new, streamlined form June 2 with which physicians can get investigational new drugs (INDs) otherwise not cleared for treatment into the hands of their patients. FDA makes no guarantees but says applicants should get their approval within 30 days -- and in some instances may get it immediately.
"As a physician, I understand the importance of being able to access investigational treatments for a patient when there are no other options to treat their serious disease or condition," said FDA Commissioner Robert Califf in announcing Form FDA 3926. He also said 3926 is "much shorter than the form previously used for individual patient expanded access requests and is designed specifically for these requests. It should take about 45 minutes for a physician to complete..."
The agency "most likely" is responding to a wave of state "right to try" laws that loosen access to experimental drugs or off-label use of drugs, says Kirstin Matthews, fellow in science and technology policy at the Center for Health and Biosciences at Rice University's Baker Institute in Houston. Such laws have been passed in 26 states; a right to try bill was just passed by the California assembly.
While "expanded access" -- FDA's preferred nomneclature to the informal terms "compassionate use" and "pre-approval access" -- is sometimes granted to patient populations, Form 3926 is to be used by physicians to apply for access to INDs for their individual patients. (Physicians previosuly used FDA 1571, an Investigational New Drug Application form that the agency now says was "not tailored to requests for individual patient expanded access.")
When it receives the 3926 application, FDA explains in related guidance, the agency must determine whether or not the patient has a "serious or immediately life-threatening disease" for which there is no acceptable alternative treatment to the IND; that the potential risks of using the IND do not outweigh the benefits; and that the patient's access will not interfere with current clinical trials.
"FDA generally intends to accept submission of a completed Form FDA 3926 to comply with the IND submission requirements" in federal law, says FDA in its guidance. The form allows the physician and patient to skip some other forms normally required for expanded access applications, such as FDA 1571 and FDA 1572.
If the situation is an emergency, physicians may submit Form 3926 by telephone, provided they get their written application in within 15 days.
Upon receipt of the form, FDA will notify the physician of its decision; if the phsyician doesn't hear within 30 days, the physician may consider the application accepted.
It's not entirely this easy: Prior to submitting the 3926, physicians are supposed to "obtain an LOA [letter of authorization] from the entity that is the sponsor of the IND." If they can't do that, they have to "contact the relevant review division at FDA to determine what information is needed to support the expanded access submission."
Also, when the drug is approved, the physician who submitted the form becomes a "sponsor-investigator" for FDA purposes and has to observe investigational drug protocols, including safety and drug disposition reports.
FDA also released with the form a Q&A on expanded access and guidance on federal regulations "regarding how patients may be charged for investigational drugs." (Note: In the latter, FDA says "it has no authority to require that the Centers for Medicare and Medicaid Services reimburse for investigational drugs for which FDA has authorized charging.")
Matthews, who is co-author of the recent study, "The Impact of Right to Try Laws on Medical Access in the United States," is happy to see the changes, but hopes for more hands-on assistance to physicians. "I would like to see FDA be pro-active in helping guide physicians and patients through to the process to make sure it is indeed easy and quick to fill out," she says. "While their Q&A forms are good, a dedicated group to answer questions from patients and physicians might also be warranted.”